Myelodysplastic syndromes current treatment algorithm 2018. From Blood Cancer Journal, Published 24 May 2018 Myelodysplastic syndromes (MDS) are defined by ineffective hematopoiesis resulting in blood cytopenias, and clonal instability with a risk of clonal evolution to acute myeloid leukemia (AML). Patients with MDS collectively have a high symptom burden and are also at risk of death from complications of cytopenias and AML. The goals of therapy for patients with MDS are to reduce disease-associated symptoms and the risk of disease progression and death, thereby improving both quality and quantity of life. Because the median age at diagnosis of MDS is ~70 years, patients frequently have comorbid conditions that may influence outcomes and treatment approaches. For instance, patients with established cardiovascular or pulmonary disease tolerate anemia poorly, while those with germline HFE mutations associated with hereditary hemochromatosis may have an elevated risk of organ toxicity from iron overload with repeated red cell transfusions. Functional defects in neutrophils or platelets may result in an infection or bleeding risk that is disproportionate to the degree of cytopenias9. Three drugs have been approved by the Food and Drug Administration (FDA) for use in MDS-related indications: the orally administered immunomodulatory drug lenalidomide, and two parenterally administered nucleoside analogs that are DNA hypomethylating agents (HMA), azacitidine, and decitabine. In addition to these agents, there is extensive off-label use of the erythropoiesis-stimulating agents (ESA) epoetin and darbepoetin in MDS, which is supported by National Comprehensive Cancer Network (NCCN) guidelines and therefore reimbursed by Medicare as a compendium use. No new drugs have been approved for MDS-related indications since decitabine’s FDA approval in 2006, underscoring the importance of clinical trial enrollment; currently only a small proportion of patients with MDS are enrolled in prospective interventional studies. #algorithm #hematology #cancer
